Are Off-Label Drugs Safe for Treating MS?

When your doctor suggests a drug to treat your multiple sclerosis (MS), your first move may be to search the internet to learn more about it. You may be surprised to find that the medicine is "off-label," which means it isn't approved to treat MS.

Is that a good idea, you wonder? The answer: It can be. A lot depends on how the medicine works and whether the benefits outweigh the risks.

Over the years, doctors have prescribed everything from cancer drugs to acne medicine to help with MS. So you have a lot of options.

When Would I Try One?

Your doctor might suggest going off-label if approved drugs aren't safe for you. You might have allergies to some medicines or find that side effects hit you too hard. Or you might have another condition that means you need to avoid certain medications.

In other cases, you might have tried the standard medications and found they didn't get the job done.

What Are the Risks?

The FDA approves drugs to treat specific conditions. Then, doctors get guidelines on when to use them, how much to give, and what side effects to look for.

When you go off-label, you're in a gray area. A cancer drug doesn't get tested in someone with MS to see if it acts any differently or to check which dose works best. So there's some extra risk there.

That said, many off-label meds for MS often have at least some research behind them. When studies show off-label drugs work for MS, they tend to have fewer risks.

Your best bet is to talk it through carefully with your doctor. Ask questions like:

  • What studies show the drug works for MS?
  • What are the side effects and risks for someone with MS?
  • Are there clinical trials I could join?

How Long Can I Use Them?

It depends on a lot of things like the dose, other conditions you have, and other medicines you take. For some drugs, there may not be enough research to know. New studies come out all the time, so check with your doctor.

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Will My Insurance Pay for It?

Not always, so it's best to find out ahead of time. Your insurance company may view it as experimental and not cover it. If that happens, you may be able to get your doctor to write a letter for you that shows what MS studies have been done with the drug.

Which Off-Label Drugs Treat MS?

You have a lot of choices. Some medicines your doctor may suggest are:

Amantadine (Osmolex ER) . It's used to treat the flu and Parkinson's disease, but it may help with fatigue from MS. It generally has few side effects and seems safe.

Azathioprine ( Imuran ) . Doctors have prescribed this drug to treat MS for over 40 years. It can cut the number of relapses you get and keep your MS from getting worse. If you use it for more than 10 years or take more than 600 grams over your lifetime, it may raise your risk of cancer.

Cyclophosphamide . Doctors sometimes recommend it for progressive forms of MS, but results have been mixed. It may slow down your disease and help with symptoms, but it can also come with side effects like nausea and vomiting that can be tough for some people.

Duloxetine ( Cymbalta , Irenka). Your doctor may suggest it to treat pain and depression from MS. It seems generally safe and without too many side effects.

Methotrexate . It can slow down progressive forms of MS and may put off problems in your shoulders, arms, and hands. One study showed that it was safe to use for at least 3 to 6 years.

Minocycline . An antibiotic originally used for acne, this drug's been around for 50 years. It looks to be helpful in slowing down relapsing-remitting MS in people who recently had their first symptoms.

Mycophenolate mofetil (CellCept). This drug may cut down on your relapses and slow down the disease. It may raise your chances of getting cancer or an infection. More research is needed to see if it's safe to take long term.

Pregabalin (Lyrica). This is another option for treating pain from MS. It looks to be safe with few side effects.

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Rituximab (Rituxan) . It can slow down relapsing-remitting MS and cut back on relapses. For most people, it doesn't seem to cause any major issues.

Simvastatin (Zocor) . It's typically used for high cholesterol, but it shows promise in slowing brain shrinkage from secondary progressive MS. And it's considered safe with few side effects.

WebMD Medical Reference Reviewed by Jennifer Robinson, MD on May 30, 2019

Sources

SOURCES:

National Multiple Sclerosis Society: "Non-approved treatments used for MS disease modification," "Amantadine," "Pain."

Multiple Sclerosis Society of Canada: "Common acne medication offers new treatment for multiple sclerosis."

U.S. National Library of Medicine: "Current and emerging therapies in multiple sclerosis: a systematic review," "Ten Common Questions (and Their Answers) About Off-label Drug Use," "Azathioprine for multiple sclerosis," "Double-Blind Controlled Randomized Trial of Cyclophosphamide versus Methylprednisolone in Secondary Progressive Multiple Sclerosis," "A Randomized Placebo-Controlled Trial of Duloxetine for Central Pain in Multiple Sclerosis," "Duloxetine is effective in treating depression in multiple sclerosis patients: an open-label multicenter study."

Biogen: "Avonex Medication Guide."

FDA: "Understanding Unapproved Use of Approved Drugs "Off Label"."

Journal of the American Medical Association: "Association of Off-label Drug Use and Adverse Drug Events in an Adult Population."

American Cancer Society: "Off-Label Drug Use."

Medscape: "Multiple Sclerosis Medication," "Cladribine Back in the Running for Multiple Sclerosis," "Cladribine (Mavenclad) for MS Approved in Europe."

Cleveland Clinic: "Azathioprine and Multiple Sclerosis."

Multiple Sclerosis Trust: "Cyclophosphamide (Endoxana)," "Modafinil (Provigil)," "Pregabalin (Lyrica)."

Partners Multiple Sclerosis Center: "Cyclophosphamide (Cytoxan)."

Tisch MS Research Center of New York: "Safety of Long-term Intrathecal Methotrexate Therapy in Progressive Forms of Multiple Sclerosis."

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